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SPK-3006

DRUG2 trials

Sponsors

Genentech Inc., Spark Therapeutics, Inc.

Conditions

Acid Maltase DeficiencyGlycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesPompe DiseasePompe Disease (Late-onset)Pompe Disease (also known as glycogen storage disease type II)

Phase 1

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Active, not recruitingNCT04093349
Spark Therapeutics, Inc.Acid Maltase Deficiency, Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II +4
Start: 2020-10-01End: 2032-04-30Updated: 2024-11-27
Phase 1/2 Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults with Late-Onset Pompe Disease
RecruitingCTIS2023-509303-32-00
Genentech Inc.Pompe Disease (also known as glycogen storage disease type II)
Start: 2021-10-19Target: 2Updated: 2025-10-21